Trial Outcomes & Findings for Omacetaxine in Patients With Intermediate-1 and Higher Risk Myelodysplastic Syndrome (MDS) Post Hypomethylating Agent (HMA) Failure (NCT NCT02159872)
NCT ID: NCT02159872
Last Updated: 2021-06-09
Results Overview
Overall survival defined as the time from treatment start to the time of death. Overall survival continuously monitored using the Bayesian method.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
48 participants
Primary outcome timeframe
Up to 2.5 Years
Results posted on
2021-06-09
Participant Flow
Recruitment Period: May 2015 to September 2018
Participant milestones
| Measure |
Omacetaxine
Omacetaxine 1.25 mg/m2 subcutaneously every 12 hours on Days 1-3 of every 28-day study cycle. Participant may continue taking the study drug for up to 24 cycles of treatment.
Omacetaxine: 1.25 mg/m2 subcutaneously every 12 hours on Days 1-3 of every 28-day study cycle.
|
|---|---|
|
Overall Study
STARTED
|
48
|
|
Overall Study
COMPLETED
|
48
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Omacetaxine in Patients With Intermediate-1 and Higher Risk Myelodysplastic Syndrome (MDS) Post Hypomethylating Agent (HMA) Failure
Baseline characteristics by cohort
| Measure |
Omacetaxine
n=48 Participants
Omacetaxine 1.25 mg/m2 subcutaneously every 12 hours on Days 1-3 of every 28-day study cycle. Participant may continue taking the study drug for up to 24 cycles of treatment.
Omacetaxine: 1.25 mg/m2 subcutaneously every 12 hours on Days 1-3 of every 28-day study cycle.
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
5 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
43 Participants
n=5 Participants
|
|
Age, Continuous
|
75.5 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
17 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
31 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
3 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
42 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
48 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Up to 2.5 YearsOverall survival defined as the time from treatment start to the time of death. Overall survival continuously monitored using the Bayesian method.
Outcome measures
| Measure |
Omacetaxine
n=48 Participants
Omacetaxine 1.25 mg/m2 subcutaneously every 12 hours on Days 1-3 of every 28-day study cycle. Participant may continue taking the study drug for up to 24 cycles of treatment.
Omacetaxine: 1.25 mg/m2 subcutaneously every 12 hours on Days 1-3 of every 28-day study cycle.
|
|---|---|
|
Overall Survival (OS)
|
7.5 Months
Interval 0.1 to 30.0
|
PRIMARY outcome
Timeframe: Up to 2 yearsResponse is Complete Response (CR) + Partial Response (PR) + Hematologic Improvement (HI). CR is the normalization of the peripheral blood and bone marrow with \</= 5% bone marrow blasts, a peripheral blood granulocyte count \>/= (1.0x10\^9/L, and a platelet count \>/= 100x10\^9/L). PR is the same as CR except for the presence of 6-15% marrow blasts, or 50% reduction if \<15% at start of treatment. HI meets all of the criteria for CR except for platelet recovery to \>/=100x10\^9L.
Outcome measures
| Measure |
Omacetaxine
n=48 Participants
Omacetaxine 1.25 mg/m2 subcutaneously every 12 hours on Days 1-3 of every 28-day study cycle. Participant may continue taking the study drug for up to 24 cycles of treatment.
Omacetaxine: 1.25 mg/m2 subcutaneously every 12 hours on Days 1-3 of every 28-day study cycle.
|
|---|---|
|
Number of Participants With a Response
|
16 Participants
|
Adverse Events
Omacetaxine
Serious events: 30 serious events
Other events: 23 other events
Deaths: 15 deaths
Serious adverse events
| Measure |
Omacetaxine
n=48 participants at risk
Omacetaxine 1.25 mg/m2 subcutaneously every 12 hours on Days 1-3 of every 28-day study cycle. Participant may continue taking the study drug for up to 24 cycles of treatment.
Omacetaxine: 1.25 mg/m2 subcutaneously every 12 hours on Days 1-3 of every 28-day study cycle.
|
|---|---|
|
Respiratory, thoracic and mediastinal disorders
Acute Respiratory Failure
|
2.1%
1/48 • Number of events 1 • Up to 4 years
|
|
Cardiac disorders
Atrial Fibrillation
|
2.1%
1/48 • Number of events 1 • Up to 4 years
|
|
Infections and infestations
Bacteremia Infection
|
2.1%
1/48 • Number of events 1 • Up to 4 years
|
|
Cardiac disorders
Cardiac Ischemia
|
2.1%
1/48 • Number of events 1 • Up to 4 years
|
|
General disorders
Chest Pain
|
2.1%
1/48 • Number of events 1 • Up to 4 years
|
|
Cardiac disorders
Congestive Heart Failure
|
2.1%
1/48 • Number of events 1 • Up to 4 years
|
|
Gastrointestinal disorders
Diarrhea
|
2.1%
1/48 • Number of events 1 • Up to 4 years
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
2.1%
1/48 • Number of events 1 • Up to 4 years
|
|
General disorders
Fatigue
|
2.1%
1/48 • Number of events 1 • Up to 4 years
|
|
General disorders
Graft Versus Host Disease
|
2.1%
1/48 • Number of events 1 • Up to 4 years
|
|
Injury, poisoning and procedural complications
Hip Fracture
|
2.1%
1/48 • Number of events 1 • Up to 4 years
|
|
General disorders
Left Shoulder Pain
|
2.1%
1/48 • Number of events 1 • Up to 4 years
|
|
Gastrointestinal disorders
Oral Mucositis
|
2.1%
1/48 • Number of events 1 • Up to 4 years
|
|
Cardiac disorders
Myocardial Infarction
|
2.1%
1/48 • Number of events 1 • Up to 4 years
|
|
Gastrointestinal disorders
Nausea & Vomiting
|
2.1%
1/48 • Number of events 1 • Up to 4 years
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Neoplasms benign, Malignant
|
2.1%
1/48 • Number of events 1 • Up to 4 years
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory Failure
|
2.1%
1/48 • Number of events 1 • Up to 4 years
|
|
Infections and infestations
Right Arm Cellulitis
|
2.1%
1/48 • Number of events 1 • Up to 4 years
|
|
Infections and infestations
Soft Tissue Infection
|
2.1%
1/48 • Number of events 1 • Up to 4 years
|
|
Nervous system disorders
Syncope
|
2.1%
1/48 • Number of events 1 • Up to 4 years
|
|
Nervous system disorders
Transient Ischemic Attack
|
2.1%
1/48 • Number of events 1 • Up to 4 years
|
|
Infections and infestations
Urinary Tract Infection
|
2.1%
1/48 • Number of events 1 • Up to 4 years
|
|
Nervous system disorders
Subaracnoid hemorrhage
|
2.1%
1/48 • Number of events 1 • Up to 4 years
|
|
Cardiac disorders
Cardiac Arrest
|
2.1%
1/48 • Number of events 1 • Up to 4 years
|
|
Respiratory, thoracic and mediastinal disorders
Hemoptysis
|
2.1%
1/48 • Number of events 1 • Up to 4 years
|
|
Nervous system disorders
Intracranial Hemorrhage
|
2.1%
1/48 • Number of events 1 • Up to 4 years
|
|
Respiratory, thoracic and mediastinal disorders
Shortness of Breath
|
4.2%
2/48 • Number of events 3 • Up to 4 years
|
|
Gastrointestinal disorders
Epigastric Pain
|
2.1%
1/48 • Number of events 1 • Up to 4 years
|
|
Blood and lymphatic system disorders
Right Cervical Lymphadenopathy
|
2.1%
1/48 • Number of events 1 • Up to 4 years
|
|
Gastrointestinal disorders
Gingival Infiltration
|
2.1%
1/48 • Number of events 1 • Up to 4 years
|
|
Infections and infestations
Cellulitis
|
6.2%
3/48 • Number of events 3 • Up to 4 years
|
|
Injury, poisoning and procedural complications
Fall
|
4.2%
2/48 • Number of events 2 • Up to 4 years
|
|
Immune system disorders
Fever, Flu Shot Reaction
|
2.1%
1/48 • Number of events 2 • Up to 4 years
|
|
Vascular disorders
Hypotension
|
4.2%
2/48 • Number of events 2 • Up to 4 years
|
|
Infections and infestations
RSV Pneumonia
|
2.1%
1/48 • Number of events 2 • Up to 4 years
|
|
Infections and infestations
Sepsis
|
4.2%
2/48 • Number of events 2 • Up to 4 years
|
|
Musculoskeletal and connective tissue disorders
Spasticity (Severe Muscle Spasm)
|
4.2%
2/48 • Number of events 2 • Up to 4 years
|
|
General disorders
Multi-Organ Failure
|
2.1%
1/48 • Number of events 1 • Up to 4 years
|
|
Infections and infestations
Lung Infection
|
6.2%
3/48 • Number of events 3 • Up to 4 years
|
|
Gastrointestinal disorders
Upper Gastrointestianl Hemorrhage
|
4.2%
2/48 • Number of events 3 • Up to 4 years
|
|
General disorders
Fever
|
10.4%
5/48 • Number of events 5 • Up to 4 years
|
|
General disorders
Death
|
14.6%
7/48 • Number of events 7 • Up to 4 years
|
|
Infections and infestations
Pneumonia
|
12.5%
6/48 • Number of events 7 • Up to 4 years
|
|
Blood and lymphatic system disorders
Neutropenic Fever
|
16.7%
8/48 • Number of events 13 • Up to 4 years
|
Other adverse events
| Measure |
Omacetaxine
n=48 participants at risk
Omacetaxine 1.25 mg/m2 subcutaneously every 12 hours on Days 1-3 of every 28-day study cycle. Participant may continue taking the study drug for up to 24 cycles of treatment.
Omacetaxine: 1.25 mg/m2 subcutaneously every 12 hours on Days 1-3 of every 28-day study cycle.
|
|---|---|
|
General disorders
Hemorrhage
|
6.2%
3/48 • Number of events 3 • Up to 4 years
|
|
Nervous system disorders
Forgetfulness
|
8.3%
4/48 • Number of events 4 • Up to 4 years
|
|
Gastrointestinal disorders
Mouth Sores
|
8.3%
4/48 • Number of events 4 • Up to 4 years
|
|
Blood and lymphatic system disorders
Neutropenic Fever
|
8.3%
4/48 • Number of events 4 • Up to 4 years
|
|
Nervous system disorders
Dizziness
|
10.4%
5/48 • Number of events 5 • Up to 4 years
|
|
General disorders
Edema
|
12.5%
6/48 • Number of events 6 • Up to 4 years
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
14.6%
7/48 • Number of events 7 • Up to 4 years
|
|
Infections and infestations
Infection
|
25.0%
12/48 • Number of events 12 • Up to 4 years
|
|
Gastrointestinal disorders
Nausea/Vomiting
|
41.7%
20/48 • Number of events 20 • Up to 4 years
|
|
General disorders
Fatigue
|
47.9%
23/48 • Number of events 23 • Up to 4 years
|
Additional Information
Elias Jabbour MD/Professor
The University of Texas MD Anderson Cancer Center
Phone: 713-792-4764
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place